• Methods Mol. Biol. · Jan 2018

    From gRNA Identification to the Restoration of Dystrophin Expression: A Dystrophin Gene Correction Strategy for Duchenne Muscular Dystrophy Mutations Using the CRISPR-Induced Deletion Method.

    • Benjamin Duchêne, Jean-Paul Iyombe-Engembe, Joël Rousseau, Jacques P Tremblay, and Dominique L Ouellet.
    • Centre de Recherche du CHU de Québec-Université Laval, 2705 Boul. Laurier, Québec, QC, Canada, G1V 4G2.
    • Methods Mol. Biol. 2018 Jan 1; 1687: 267-283.

    AbstractThe discovery of the CRISPR-Cas9 system raises hope for the treatment of many genetic disorders. We describe here an approach based on the use of a pair of single guide RNAs to form a hybrid exon that does not only restore the dystrophin gene reading frame but also results in the production of a dystrophin protein with an adequate structure of the central rod-domain, with a correct spectrin-like repeat. The therapeutic approach described here involved DMD patient cells having a deletion of exons 51-53 of the DMD gene.

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